Following our summer break, we are resuming coverage of Novartis (NYSE: NVS ) ( OTCPK:NVSEF ). Although Trump enacted a 39% duty on Swiss goods in early August, the measure spares pharmaceuticals, given the USA's reliance on SwissBuy-side hedge professionals conducting fundamental, income oriented, long term analysis across sectors globally in developed markets. Please shoot us a message or leave a comment to discuss ideas.DISCLOSURE: All of our articles are a matter of opinion, informed as they might be, a ...

撰文丨王聪 编辑丨王多鱼 排版丨水成文 肌萎缩侧索硬化症 （ALS） ，也叫做 渐冻症 ，是一种进行性神经退行性疾病，其特征是运动皮层和脊髓中的运动神经元丧失。ALS 和 额颞叶痴呆 （FTD） 最 常见的遗传病因是 C9orf72 基因第一个内含子中 GGGGCC （G 4 C 2 ） 重复序列的扩增。这一扩增促进神经退行性病变的机制尚不清楚，但有研究证据表 明，扩增重复序列的转录和翻译导致了毒性功能增益。正义链 G4C2 或反义链 C4G2 重复 RNA 可以捕获 RNA 结合蛋白，从而破坏其功能。含重复序列的 mRNA 的翻译会产生二肽重复蛋白 （DPR） ，这些蛋白已被证明可通过多种机制发挥毒性作用。 反义寡核苷酸 （ASO） 药物在杜氏肌营养不良 （DMD） 等疾病中获得了成功，受此激励，ASO 已成为 C9orf72 相关肌萎缩侧索硬化症 （c9ALS） 的主要治 疗策略。 BIIB078 是 Biogen 和 Ionis 公司合作开发的一款用于治疗 c9ALS 的 ASO 药物，旨在靶向 C9orf72 基因第一个内含子中的 18 个碱基对序列，并通过 RNase- H 切割降解含 G4C2 重 ...

近日，CDE官网显示，山东新时代药业有限公司按4类化药注册申报的尼可地尔片上市申请申请已获受理。作为上市40余年的经典抗心绞痛药，尼可地尔院 内年销售额超14亿元，"老药翻红"成为新一代"卷王"，并成功入围新国采。 | 受理品种目录浏览 | 在审品种目录浏览 | | | | | | | | --- | --- | --- | --- | --- | --- | --- | --- | | 2025 | ▼ | 药品类型: | 全部 | レ 申请类型: | 全部 | ਾ | 童询 | | 请输入受理号 | | 药品名称: | 尼可地尔片 | 企业名称: | 请输入企业名称 | | | | 受理号 CYHS2503128 | | 药品名称 尼可地尔片 | 药品类型 化药 | 申请类型 仿制 | 注册分类 ব | 企业名称 山东新时代药业有限公 司;山东新时代药业有 限公司: | 承办日期 2025-08-26 国际军区 | | 年 会睡着: 序号 | | | | | | | | 截图来源：CDE官网 尼可地尔（Nicorandil）作为一类兼具硝酸酯样作用和ATP敏感性钾通道（KATP）开放作用的双重机制药物，用 ...

Group 1 - The article discusses Zymeworks Inc. (NASDAQ: ZYME) and highlights its recent progress, particularly focusing on two key milestones related to its drug Zanidatamab [2] - The author operates the Biotech Analysis Central service, which provides in-depth analysis of various pharmaceutical companies and includes a model portfolio of small and mid-cap stocks [2] - The service offers a subscription model with a promotional two-week free trial and a discounted annual plan [1] Group 2 - The article does not provide specific financial data or performance metrics for Zymeworks Inc. or its products [4] - There are no disclosures regarding stock positions or business relationships with the companies mentioned, indicating an unbiased perspective [3]

Financial Performance - Group revenue declined by 360% year-over-year to $630 million due to US health program budget pressures, regulatory challenges, and evolving market dynamics[23, 29] - Gross profit decreased by 385% year-over-year to $329 million[29] - The company recognized a non-cash impairment of $465 million on US intangibles[23] - EBITDA was down by $139 million year-over-year, reaching ($107 million)[29] Strategic Decisions and Capital Position - The ANZ business was divested post year-end to Jonas Software for $350 million, with potential earn-outs of up to $735 million over three years[23] - As of July 9, the company had a net cash position of $165 million, excluding an $8 million holdback, and all debt was extinguished[23] - The company withdrew its FY26 revenue guidance due to prevailing short-term market uncertainty in the US[24] US Market Operations - US revenue declined by 360% year-over-year, mirroring the group's overall revenue decrease[39] - THRiV-powered programs contributed 31% of US revenue in FY25, exceeding the PCP (Prior Corresponding Period)[40] - The US program pipeline is valued at US$125 million (unweighted) heading into 1H FY26[24, 58] - Operating costs for continuing operations are expected to decrease by 10% in FY26 compared to FY25 due to right-sizing efforts[24, 59]

Summary of Syndax Pharmaceuticals (SNDX) Conference Call - September 02, 2025 Company Overview - **Company**: Syndax Pharmaceuticals - **Products**: Revuforj (for KMT2A-rearranged acute leukemia) and Ictimo (for chronic graft-versus-host disease, cGVHD) - **Recent Achievements**: Two rapid approvals in succession, with a focus on addressing high unmet medical needs in oncology Key Points on Revuforj - **Launch Performance**: Revuforj has seen a **43% growth in net sales** with over **$50 million** generated since launch, treating over **500 patients** [3][5] - **Market Dynamics**: - Targeting **KMT2A-rearranged patients**, with a total addressable market (TAM) of **2,000 new patients annually** [5][6] - Currently treating **25%** of the available market, with expectations to increase to **50%** by year-end [5][6] - **Treatment Duration**: Average treatment duration expected to increase from **4-6 months** to **9 months** as more patients are treated earlier in their disease progression [9][10] - **Patient Demographics**: Shift from treating late-line patients to earlier-line patients, with **70%** of current patients being in the second or third line of treatment [7][12] - **Transplant Dynamics**: Approximately **33%** of patients are expected to go for transplant, higher than the **25%** seen in clinical trials [7][8] Upcoming Regulatory Milestones - **sNDA Submission**: An sNDA for Revuforj is under review with a PDUFA date of **October 25, 2025** [21][22] - **Market Expansion**: The NPM1-mutated patient market is larger than KMT2A, with an estimated **4,500 patients** [23][24] Key Points on Ictimo - **Launch Success**: Ictimo has generated nearly **$50 million** in sales in its first full quarter, with **700 patients** treated [42][43] - **Patient Retention**: **80-90%** of patients are remaining on the drug, indicating strong tolerability [42] - **Market Coverage**: Over **80%** of transplant centers have utilized Ictimo, with a majority using it multiple times [43][44] - **Partnership with Incyte**: Co-promotion with Incyte has been efficient, leveraging their existing infrastructure [44] Future Development Plans - **Subcutaneous Formulation**: Development of a subcutaneous formulation for Ictimo is underway, which could enhance patient convenience [48] - **Clinical Trials**: Ongoing studies in combination with dexamethasone and Jakafi for earlier lines of therapy in cGVHD [49][50] Financial Outlook - **Profitability Goals**: Syndax aims to reach profitability without needing additional financing, with operating expenses expected to remain flat through **2025** [61][62] - **Collaboration Revenue**: Ictimo is already profitable on a contribution profit basis, with **$9 million** in collaboration revenue reported [61] Competitive Landscape - **Market Position**: Syndax is positioned as the only approved menin inhibitor, with a focus on maintaining a competitive edge through efficacy and multiple indications [30][40] Conclusion - **Strategic Focus**: Syndax is concentrating on successfully commercializing Revuforj and Ictimo, advancing clinical development, and achieving profitability before considering new product investments [68][69]

TEL AVIV, Israel, Sept. 02, 2025 (GLOBE NEWSWIRE) -- Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) today announced that Richard Francis, Teva's President and CEO, will present at two upcoming investor conferences in September as follows: Morgan Stanley 23rd Annual Global Healthcare ConferenceTuesday, September 9, 2025, at 8:30 A.M. ET BofA Global Healthcare Conference 2025 (London)Wednesday, September 24, 2025, at 9:00 A.M. British Summer Time (4.00 A.M. Eastern Time) To access a live webcast of ...

Core Insights - Plus Therapeutics, Inc. is a clinical-stage pharmaceutical company focused on developing targeted radiotherapeutics for central nervous system cancers [2] - The company will participate in the H.C. Wainwright 27th Annual Global Investment Conference from September 8 to 10, 2025 [1] - Dr. Marc H. Hedrick, President and CEO of Plus Therapeutics, will engage in a fireside chat at the conference, available on demand for registered investors starting September 5 [2] Company Overview - Plus Therapeutics is headquartered in Houston, Texas, and specializes in targeted radiotherapeutics aimed at difficult-to-treat CNS cancers [2] - The company is advancing a pipeline of product candidates, with lead programs targeting leptomeningeal metastases and recurrent glioblastoma [2] - Plus Therapeutics has established a supply chain through strategic partnerships to support the development, manufacturing, and potential commercialization of its products [2]

PresentationAt this time, I would like to turn the call over to Wade Walke, Senior Vice President of Investor Relations, to lead off the call. Please begin.Good morning, and welcome to Ionis Conference Call to discuss Olezarsen CORE and CORE2 top line data. As a reminder, this call is being recorded.D. WalkeSenior Vice President of Investor Relations Thank you, Ludy, and thank you to everyone who has joined us today as we discuss the groundbreaking top line results from the landmark CORE and CORE2 studies o ...

Core Viewpoint - A shareholder class action lawsuit has been filed against Biohaven Ltd. alleging misleading statements regarding the company's business and drug prospects [1] Group 1: Allegations in the Lawsuit - The lawsuit claims that Biohaven overstated the regulatory prospects of troriluzole as a treatment for spinocerebellar ataxia [1] - It is alleged that the efficacy and clinical prospects of BHV-7000 for bipolar disorder were also overstated [1] - The lawsuit suggests that the revelation of these issues could significantly negatively impact Biohaven's business and financial condition [1] Group 2: Shareholder Information - Shareholders who purchased Biohaven shares between March 24, 2023, and May 14, 2025, and experienced significant losses are encouraged to discuss their legal rights [2] - The deadline for shareholders to request to be appointed as lead plaintiff in the case is September 12, 2025 [3] - Holzer & Holzer, LLC is noted for its representation of shareholders in litigation and has recovered hundreds of millions for victims of corporate misconduct [3]